Alnylam Announces Collaboration with Novartis to Explore Targeted Therapy to Restore Liver Function

– Agreement will Bring Alnylam’s Proprietary siRNA Technology to Bear on Liver Target Identified by Novartis Researchers –

– Alnylam and Novartis have Agreed to Collaborate on Discovery and Development of siRNA-based Targeted Therapy to Restore Functional Liver Cells in Patients with End-Stage Liver Diseases --

– Collaboration Aims to Develop Liver-Targeted Therapy as a Potential Alternative to Transplantation for Patients Experiencing Liver Failure –

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced a collaboration with Novartis to leverage Alnylam’s proven, proprietary siRNA technology to inhibit a target discovered at the Novartis Institutes for BioMedical Research, potentially leading to development of a treatment designed to promote the regrowth of functional liver cells and to provide an alternative to transplantation for patients with liver failure.

“We are so pleased to collaborate with Novartis,” said Kevin Fitzgerald, Ph.D., Chief Scientific Officer at Alnylam. “We believe collaborations like this serve as an excellent example of how Alnylam's leadership in RNAi can fuel new frontiers of medicine with highly innovative targets coming from some of the most admired pharmaceutical companies.”

End-stage liver disease (ESLD) is a progressive illness, most often resulting from cirrhosis, that is characterized by the destruction of healthy liver tissue and the loss of critical liver function.¹ The disease has a profound impact on patients’ quality of life, and accounts for over one million deaths globally each year. Currently, liver transplantation is the only treatment for ESLD, but transplants are invasive procedures and there is a limited supply of organs available for patients in need. A significant need exists for medicinal alternatives to transplantation that regenerate liver tissue and restore the essential metabolic and synthetic processes that are managed by the liver.

“There remains an enormous unmet need for new types of medicines to address end-stage liver disease,” said Jay Bradner, President of the Novartis Institutes for BioMedical Research. “Building on a legacy of leadership in regenerative medicine, we have devised a restorative strategy that could potentially deliver a transformative benefit to patients with liver failure. We’re delighted now to work alongside Alnylam in this new collaboration, as the Alnylam siRNA platform is optimally suited to translate this concept to clinical investigation.”

During the exclusive three-year research collaboration, Alnylam will develop and test potential siRNAs using target-specific assays developed by Novartis. Once a lead candidate is identified, further development and clinical research will be conducted by Novartis.

About Alnylam RNAi Therapeutics

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, Alnylam developed a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO® (lumasiran), as well as Leqvio® (inclisiran), with Alnylam’s partner Novartis, which has obtained global rights to develop, manufacture and commercialize inclisiran under a license and collaboration agreement. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam, on LinkedIn, or on Instagram.

Alnylam Forward Looking Statements

Various statements in this release concerning Alnylam’s expectations, plans, aspirations, and goals, including those related to the goals of Alnylam’s collaboration with Novartis and the potential development of a treatment designed to promote the regrowth of functional liver cells and to provide an alternative to transplantation for patients with liver failure, Alnylam’s aspiration to become a leading biotech company, and the planned achievement of its “Alnylam P⁵x25” strategy, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation: the direct or indirect impact of the COVID-19 global pandemic or any future pandemic on Alnylam’s business, results of operations and financial condition and the effectiveness or timeliness of Alnylam’s efforts to mitigate the impact of the pandemic; the potential impact of the recent leadership transition on Alnylam’s ability to attract and retain talent and to successfully execute on its “Alnylam P5x25” strategy; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates, including as part of its collaboration with Novartis; the pre-clinical and clinical results for its product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling its approved products globally; delays, interruptions or failures in the manufacture and supply of its product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to successfully expand the indication for ONPATTRO (and potentially vutrisiran, if approved) in the future; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam’s ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products, including Novartis, Regeneron and Vir; the outcome of litigation; the potential impact of a current government investigation and the risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in its other SEC filings. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

References

Potosek, Jamie et al. Integration of palliative care in end-stage liver disease and liver transplantation. Journal of Palliative Medicine. 2014;17(11):1271-1277. doi:10.1089/jpm.2013.0167

Asrani SK, Devarbhavi H, Eaton J, Kamath PS. Burden of liver diseases in the world. J Hepatol. 2019 Jan;70(1):151-171. doi: 10.1016/j.jhep.2018.09.014. Epub 2018 Sep 26. PMID: 30266282.

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